Pediatric Blood & Cancer最新文献

{"title":"Systemic EBV-positive T-cell lymphoma of childhood with unusual cytogenetic findings and family history of Still's disease-associated recurrent macrophage activation syndrome","authors":"Adam Jimenez, Luis F. Carrillo, Eric D. Carlsen","doi":"10.1002/pbc.31291","DOIUrl":"10.1002/pbc.31291","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142056288","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Barriers to access of precision guided therapies for children with high-risk cancer","authors":"Carolyn Mazariego,&nbsp;Rebecca Daly,&nbsp;Brittany McGill,&nbsp;Lauren Kelada,&nbsp;Skye McKay,&nbsp;Kate Hetherington,&nbsp;David S. Ziegler,&nbsp;Claire E. Wakefield,&nbsp;Natalie Taylor","doi":"10.1002/pbc.31147","DOIUrl":"10.1002/pbc.31147","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Accessing compassionate access schemes to obtain novel therapeutic agents for children with hard-to-treat cancers can be fraught with challenges such as regulatory barriers and limited resources. This study aimed to explore clinician perspectives on the barriers, impacts and ethical considerations of accessing novel therapeutic agents within the context of a paediatric oncology precision medicine trial.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We gathered data from 37 semi-structured interviews with paediatric oncologists participating in the PRecISion Medicine for Children with Cancer (PRISM) study, a precision medicine clinical trial in Australia. The interviews, conducted over 2 years, focused on paediatric oncologist's experiences with the PRISM trial. Interviews were re-analysed to identify themes related to access pathways and any challenges in obtaining novel agents through thematic analysis. The resulting thematic framework was discussed and refined by a multidisciplinary team.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Three main themes were identified: (i) barriers to access, including poor drug availability, lack of evidence and the time burden of the application process; (ii) impacts of inaccessibility, encompassing medical consequences and financial burden on families; and (iii) ethical considerations, centred around balancing realistic expectations and providing compassionate care to patients and families. Paediatric oncologists expressed frustration with the complex regulatory landscape and the lack of systematic reporting on applications and outcomes of obtaining novel agents. Lengthy wait times for decision notifications were also highlighted, raising concerns about missed therapeutic opportunities for patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>This study provides insight to the challenges faced when seeking access to novel therapies for paediatric oncology patients. There is a clear need for improved communication, streamlining processes and increased resources to facilitate access to novel agents. Further resource development is necessary to address these complexities in accessing novel therapy agents to ultimately ensure equitable and timely access.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31147","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142056250","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Venous thromboembolism in transgender and gender non-binary youth is rare and occurs in the setting of secondary risk factors: A retrospective cohort study","authors":"Charumathi Baskaran,&nbsp;Stephanie A. Roberts,&nbsp;Ellis Barrera,&nbsp;Sarah Pilcher,&nbsp;Riten Kumar","doi":"10.1002/pbc.31284","DOIUrl":"10.1002/pbc.31284","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>The risk of venous thromboembolism (VTE) with gender-affirming hormone therapy (GAHT) in transgender and gender non-binary (TNB) youth is unclear.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>To identify the rate of VTE in a cohort of TNB youth followed in the transgender health clinic at Boston Children's Hospital, and to investigate the impact of congenital thrombophilia diagnosis on the use of GAHT.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>ICD-9 and ICD-10 codes were used to identify eligible individuals, defined as (i) having a diagnosis of gender dysphoria and (ii) venous thromboembolism (VTE). Data were abstracted from a review of medical records. A second data query assessed TNB individuals who had an associated thrombophilia diagnosis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The primary analysis included 1860 individuals. Total 942 individuals (50.6%) had started GAHT at the time of data analysis. Mean age (±SD) at GAHT initiation was 16.8 (±1.9) years. Five thrombotic events were identified in three (0.13%) individuals, all in the setting of additional VTE risk factors. Only two of five thrombotic events occurred while receiving GAHT. The rate of VTE in the GAHT cohort did not statistically differ from the rate of VTE in the non-GAHT cohort (0.1% vs. 0.2%, <i>p</i> = .62). Of the 10 individuals diagnosed with a congenital thrombophilia, two transmasculine individuals received prophylactic anticoagulation prior to GAHT. No VTE has been reported to date in this cohort.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>In our cohort, VTE was rare in the TNB youth and was not associated with GAHT use. TNB youth with congenital thrombophilia have not developed VTE in the setting of GAHT use to date.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142073467","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Surgical management of rare tumors (Part 1).","authors":"Alyssa Stetson, Saurabh Saluja, Danielle B Cameron, Sara A Mansfield, Stephanie F Polites, Joshua N Honeyman, John P Dahl, Mary T Austin, Jennifer H Aldrink, Emily R Christison-Lagay","doi":"10.1002/pbc.31287","DOIUrl":"https://doi.org/10.1002/pbc.31287","url":null,"abstract":"<p><p>With an annual cumulative occurrence of approximately 15,000 in North America, all childhood cancers are rare. Very rare cancers as defined by both the European Cooperative Study Group for Rare Pediatric Cancers and the Children's Oncology Group fall into two principal categories: those so uncommon (fewer than 2 cases/million) that their study is challenging even through cooperative group efforts (e.g., pleuropulmonary blastoma and desmoplastic small round cell tumor) and those that are far more common in adults and therefore rarely studied in children (e.g., thyroid, melanoma, and gastrointestinal stromal tumor). Treatment strategies for these latter tumors are typically based on adult guidelines, although the pediatric variants of these tumors may harbor different genetic signatures and demonstrate different behavior. If melanoma and differentiated thyroid cancer are excluded, other rare cancer types account for only 2% of the cancers in children aged 0 to 14. This article highlights several of the most common rare tumor types.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142056287","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Attention and executive functioning in children and adolescents treated for high-risk acute lymphoblastic leukemia: A report from the Children's Oncology Group (COG)","authors":"Kristina K. Hardy,&nbsp;Leanne Embry,&nbsp;John A. Kairalla,&nbsp;Christina Sharkey,&nbsp;Anthony R. Gioia,&nbsp;Danielle Griffin,&nbsp;Carly Berger,&nbsp;Hannah S. Weisman,&nbsp;Robert B. Noll,&nbsp;Naomi J. Winick","doi":"10.1002/pbc.31179","DOIUrl":"10.1002/pbc.31179","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>Survivors of childhood B-acute lymphoblastic leukemia (B-ALL) are at risk for difficulties with attention and executive functioning (EF) as a late effect of treatment. The present study aimed to identify treatment and demographic factors associated with risk for difficulties with EF in youth treated for high-risk B-ALL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Method</h3>\u0000 \u0000 <p>Children and adolescents with B-ALL treated on Children's Oncology Group (COG) protocol AALL0232 were randomized to high-dose or escalating-dose methotrexate (MTX), and either dexamethasone or prednisone during the induction phase. Neuropsychological functioning was evaluated via protocol AALL06N1, including performance-based and parent-report measures, for 177 participants (57% female, 81% white; mean age at diagnosis = 8.4 years; SD = 5.0) 8-24 months following treatment completion.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Mean scores for all attention and EF measures were within the average range, with no significant differences as a function of MTX delivery or steroid treatment (all <i>p</i> &gt; 0.05). In multivariable models, participants with US public insurance exhibited significantly greater parent-reported EF difficulties than those with US private or non-US insurance (<i>p</i> ≤ 0.05). Additionally, participants diagnosed under 10 years of age performed significantly more poorly on measures of attention (i.e., continuous performance task, <i>p</i> ≤ 0.05) and EF (i.e., verbal fluency and tower planning task, <i>p</i> ≤ 0.05).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>For survivors of pediatric B-ALL, treatment-related factors were not associated with attention or EF outcomes. In contrast, outcomes varied by demographic characteristics, including age and insurance type, an indicator of economic hardship. Future research is needed to more directly assess the contribution of socioeconomic status on cognitive outcomes in survivors.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31179","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142036605","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dyadic coping experiences of parents of children with vascular anomalies","authors":"Anna M. Kerr,&nbsp;Jacqueline Wehrli,&nbsp;Clarice Contente,&nbsp;Prathana D.,&nbsp;Bryan A. Sisk","doi":"10.1002/pbc.31261","DOIUrl":"10.1002/pbc.31261","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Vascular anomalies (VAs) are a spectrum of rare pediatric disorders that require coordinated care from multiple subspecialists. Parents often struggle to coordinate care for their child's complex rare disorder. Even when they do access expert care, parents of children with VAs report high levels of stress and uncertainty. While previous research has explored parents’ experiences navigating care for VAs, we know very little about how parents cope with stress together. Given the effect that dyadic coping can have on individual, couple, family, and child outcomes, we aimed to gain a better understanding of dyadic coping in the context of VAs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedures</h3>\u0000 \u0000 <p>We collected data using semi-structured interviews with 27 parents (13 dyads and one individual parent). Data were analyzed using dyadic thematic analysis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Parents experienced stress related to medical, personal, logistical, and financial aspects of their child's healthcare. They relied on eight coping strategies: active coping, seeking emotional support, seeking informational support, cognitive avoidance, distraction, cognitive reframing, acceptance, and internalization. When analyzed together, we found evidence of five dyadic coping dynamics: collaborative, supportive, delegated, separate, and negative.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Dyadic coping is complex and multilayered for parents of children with VAs. While the child's diagnosis is considered a shared stressor, both parents may not share preferred coping strategies. Parents of the same child may also be coping with different medical, relational/social, personal, or logistical stressors altogether. Psychosocial interventions designed to facilitate parental coping should address these complex coping dynamics.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31261","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142018296","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High-throughput sequencing of archival cerebrospinal fluid specimens defines B-lymphoblastic leukemia clonal composition","authors":"Karl Foley,&nbsp;Diana G. Adlowitz,&nbsp;Cameron Baker,&nbsp;Philip J. Rock,&nbsp;Richard Burack,&nbsp;Carol Fries","doi":"10.1002/pbc.31281","DOIUrl":"10.1002/pbc.31281","url":null,"abstract":"<p>Detailed characterization of the B-lymphoblastic leukemia (B-ALL) cells which invade the central nervous system (CNS) has been limited by practical challenges. To test whether the clonal composition of the cerebrospinal fluid (CSF) reflects the primary B-ALL tissue, we applied immunoglobulin (Ig) high-throughput sequencing (HTS) of archival CSF cytospin preparations from six patients with morphologically defined CNS involvement. We discovered that most CSF clones are detectable at some timepoint in the primary tissue, but that shifting clonal abundance is prevalent across tissue sites between diagnosis and relapse. Ig HTS of CSF cytospins may improve understanding of sanctuary site dissemination in B-ALL.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142018297","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sirolimus for vascular anomalies associated with PTEN hamartoma tumor syndrome","authors":"Alexandra Zabeida,&nbsp;Jack J. Brzezinski,&nbsp;Jonathan D. Wasserman,&nbsp;Cheryl Cytrynbaum,&nbsp;Rosanna Weksberg,&nbsp;Kelley Zwicker,&nbsp;Kevin Zbuk,&nbsp;Alessandro Gasparetto,&nbsp;Laura Willis,&nbsp;Michelle Fantauzzi,&nbsp;Manuel Carcao","doi":"10.1002/pbc.31282","DOIUrl":"10.1002/pbc.31282","url":null,"abstract":"<p>Phosphatase and tensin homolog (<i>PTEN</i>) hamartoma tumor syndrome (PHTS) is a rare condition associated with vascular anomalies and increased tumor risk. Sirolimus, an mTOR inhibitor used for managing vascular anomalies is underexplored in PHTS. A single-institution retrospective review of children with PHTS and vascular anomalies treated with sirolimus identified seven patients. Median age at sirolimus initiation was 10 years. After a median 2.5-year follow-up, six of seven patients (86%) showed significant clinical improvement. No significant adverse effects were observed, except mild buccal ulcers and acne. This study supports sirolimus as an effective and safe treatment for vascular anomalies in a small group of children with PHTS.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142009233","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A diagnostic challenge: Leukemia cutis masquerading as infantile hemangioma","authors":"Joe Khodeir,&nbsp;Paul Ohanian,&nbsp;Hala Abi Rached Megarbane","doi":"10.1002/pbc.31279","DOIUrl":"10.1002/pbc.31279","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141996247","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exploring the role of pain on physical activity among youth with acute lymphoblastic leukemia using the biopsychosocial model","authors":"Anna E. van Asselt,&nbsp;Robert C. Gibler,&nbsp;Meghan Tokala,&nbsp;Meredith L. Dreyer Gillette,&nbsp;Kimberly L. Klages,&nbsp;Renee Gilbert,&nbsp;Jacee Weber,&nbsp;Carolyn R. Bates","doi":"10.1002/pbc.31276","DOIUrl":"10.1002/pbc.31276","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Engaging in physical activity (PA) throughout cancer treatment offers many benefits, but may be challenging due to cancer-related pain. Pain research in pediatric cancer has primarily focused on procedural pain, with fewer studies exploring how pain affects PA. The current study qualitatively investigated the impact of pain on PA in youth with acute lymphoblastic leukemia (ALL) using a biopsychosocial framework.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>As part of a larger study, caregivers (<i>N </i>= 17) of a child diagnosed with ALL and on treatment for less than 1 year completed a semi-structured interview about perceptions of their child's health behaviors during ALL treatment. This secondary analysis focused specifically on discussions about pain and its impact on PA. We followed Braun and Clarke's (2006) six-step thematic analysis framework to identify themes of pain-related barriers to PA.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Key pain-related barriers endorsed by caregivers included: interactions among pain and treatment effects, caregiver distress around seeing their child in pain, and fear of interfering with medical equipment. Despite these barriers, caregivers found creative solutions to adapt activities for their child. Caregivers were reassured by PA advice from their medical team; however, advice varied between teams.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The relationship between pain and PA during ALL treatment is influenced by an intricate system of biological (e.g., treatment effects), psychological (e.g., parental distress), and social (e.g., communication among families and medical teams) factors. Future directions include identifying evidence-based PA recommendations and exploring family–team communication dynamics. This study also highlights a need to prioritize ALL pain management and involve caregivers in behavioral treatment protocols to improve PA.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4,"publicationDate":"2024-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141996248","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}